NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

An­oth­er day, an­oth­er xeno­trans­plant, as Unit­ed Ther­a­peu­tics looks to beat com­peti­tors to sci-fi-es­que break­through

Xeno­trans­plan­ta­tion is hav­ing a mo­ment.

Last Oc­to­ber, a team from NYU suc­cess­ful­ly trans­plant­ed a kid­ney from a pig in­to a brain-dead pa­tient, al­though ob­servers cast doubt on the im­por­tance of the ex­per­i­ment. Then, ear­li­er this month, sur­geons at the Uni­ver­si­ty of Mary­land trans­plant­ed a pig heart in­to a dy­ing hu­man, who ap­pears to still be sta­ble.

Jayme Locke

Now, an­oth­er group is plant­i­ng a flag in the xeno­trans­plan­ta­tion field. Sur­geons at the Uni­ver­si­ty of Al­aba­ma at Birm­ing­ham said Thurs­day they have achieved the first kid­ney trans­plant from a pig to a brain-dead pa­tient, pub­lish­ing their peer-re­viewed find­ings on­line. The team, aim­ing to dif­fer­en­ti­ate it­self from the oth­ers through the ge­net­ic mod­i­fi­ca­tions used, is hop­ing there’s now enough re­search to soon be­gin clin­i­cal xeno­trans­plan­ta­tion stud­ies.

“We have bridged crit­i­cal knowl­edge gaps and ob­tained the safe­ty and fea­si­bil­i­ty da­ta nec­es­sary to be­gin a clin­i­cal tri­al in liv­ing hu­mans with end-stage kid­ney fail­ure dis­ease,” said Jayme Locke, di­rec­tor of the UAB surgery de­part­ment’s trans­plan­ta­tion in­sti­tute, in pre­pared re­marks.

The com­mon thread through all these trans­plan­ta­tion ef­forts? That would be Unit­ed Ther­a­peu­tics’ Re­vivi­cor, which sup­plied the or­gans to each of the aca­d­e­m­ic teams in ques­tion. The biotech’s ge­net­i­cal­ly al­tered pigs were ap­proved by the FDA in De­cem­ber 2020 for use in food and po­ten­tial ther­a­peu­tic us­es.

Each of the Re­vivi­cor or­gans used con­tained ge­net­ic mod­i­fi­ca­tions, but the NYU pro­ce­dure on­ly in­clud­ed the elim­i­na­tion of a cer­tain sug­ar from the or­gan’s sur­face, with the goal of re­duc­ing the body’s re­jec­tion re­sponse. The Mary­land and UAB teams, mean­while, used or­gans con­tain­ing at least 10 dif­fer­ent ge­net­ic mod­i­fi­ca­tions.

Like the Mary­land team, UAB re­searchers trans­plant­ed the or­gan in­side the pa­tient’s body, where­as NYU on­ly su­tured the kid­ney to the pa­tient’s leg. UAB, though, con­duct­ed its pro­ce­dure on a brain-dead pa­tient as NYU did, while Mary­land trans­plant­ed the pig heart in­to a ter­mi­nal­ly ill man.

Giv­en the mas­sive short­age of avail­able or­gans for pa­tients who need trans­plants, sci­en­tists have for years the­o­rized how to safe­ly and ef­fec­tive­ly make xeno­trans­plan­ta­tion work. In ad­di­tion to Re­vivi­cor, bio­phar­mas such as Tec­tor, eGe­n­e­sis and Qi­han Biotech are work­ing to de­vel­op ready-made or­gans.

In par­tic­u­lar, eGe­n­e­sis proved the ear­ly VC fa­vorite, win­ning back­ing from promi­nent firms like Bay­er’s in­vest­ment arm and Far­al­lon, in ad­di­tion to Har­vard. But the com­pa­ny is still wait­ing to hold a pre-IND meet­ing with the FDA, eGe­n­e­sis spokesper­son David Carmel told End­points News. And though eGe­n­e­sis has ac­quired the nec­es­sary pathogen-free fa­cil­i­ty that Carmel said meets all FDA cri­te­ria, reg­u­la­tors have yet to in­spect it and like­ly won’t do so un­til the com­pa­ny is ready to make a prod­uct that can be put in­to hu­mans.

Kid­neys in par­tic­u­lar could be a po­ten­tial gold mine for com­pa­nies able to breed ge­net­i­cal­ly mod­i­fied pigs and a re­lief for pa­tients and their fam­i­lies. More than 800,000 Amer­i­cans are liv­ing with kid­ney fail­ure and most nev­er re­ceive a donor’s kid­ney. That can re­sult in in­ten­sive and ex­cru­ci­at­ing dial­y­sis treat­ments, sharply re­duc­ing pa­tients’ qual­i­ty of life.

Up­date: This ar­ti­cle has been up­dat­ed with com­ment from eGe­n­e­sis.

Bio­mark­er 'roadmap­s' and the fu­ture of can­cer R&D; Cur­tain rais­es on #AS­CO22; Pfiz­er, No­var­tis tack­le drug ac­cess; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

While this was not a week for earth-shattering news, there were certainly a lot of interesting tidbits. If you found this recap helpful, please recommend it to your friends and colleagues. We’ll see you on the other side of the long weekend.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,800+ biopharma pros reading Endpoints daily — and it's free.

Keep­ing pres­sure on Am­gen, Mi­rati draws mixed re­views on lat­est cut of KRAS da­ta

As the close runner-up to Amgen’s Lumakras in the KRAS race, any data cut from Mirati’s adagrasib continues to draw scrutiny from analysts. And the latest batch of numbers from ASCO is a decidedly mixed bag.

While a quick comparison suggests that adagrasib spurred slightly more responses and led to a longer overall survival than Lumakras among a group of non-small cell lung cancer patients, its duration of response appears shorter and the safety profile continues to spark concern.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,800+ biopharma pros reading Endpoints daily — and it's free.

Ann is one of ViiV Healthcare's newest spokespeople as the retired school administrator speaks up about her HIV status.

GSK's Vi­iV de­buts next evo­lu­tion in HIV med Dova­to cam­paign with new spokes­peo­ple and new mes­sage

When Ann saw the first TV commercials for HIV medicine Dovato, she didn’t see herself represented. So the 74-year-old retired school administrator who’s been living with HIV since 1998, reached out to GSK’s ViiV Healthcare and asked why not?

Now Ann is one of three people starring in ViiV’s latest Dovato campaign called “Detect This.” The next-step evolution in the branded campaign plays on the word “detect” — often used in describing HIV status under control as undetectable — but in this case, uses the word as a directive for people to understand they can use fewer medicines.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,800+ biopharma pros reading Endpoints daily — and it's free.

Switzer­land to de­stroy over 600,000 ex­pired dos­es of Mod­er­na Covid vac­cine

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

Lina Khan, FTC chair (Graeme Jennings/Pool via AP Images)

Pile-on over PBMs con­tin­ues with FTC com­ments and a new bi­par­ti­san Sen­ate bill

More than 500 stakeholders sent comments to the FTC on whether the commission should look further into pharma middlemen, known as PBMs, with many of the commenters calling for more federal oversight.

Similar to the critical open comment period in a deadlocked FTC session last February, pharmacies and pharmacy groups are continuing to call out the lack of transparency among the top 3 PBMs, which control about 80% of the market.

Pharma brands are losing their shine with US consumers who are now thinking about the economy and inflation instead of Covid. (Credit: Shutterstock)

Phar­ma brands fade in an­nu­al Har­ris con­sumer vis­i­bil­i­ty poll: Mod­er­na drops off and Pfiz­er dips

As Covid-19 concerns are fading in the US, so is biopharma visibility. The annual Axios Harris Poll survey to determine and rank the 100 most top-of-mind brands in the US finds Moderna, which was No. 3 last year, not on the list at all for 2022, and Pfizer sinking 37 spots.

However, it’s not that Moderna or Pfizer did anything wrong, it’s just that Americans have moved on to other worries beyond Covid.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,800+ biopharma pros reading Endpoints daily — and it's free.

HHS Secretary Xavier Becerra (Jacquelyn Martin/AP Images)

HHS fin­ish­es off Trump-era rule that would've erased ba­sic FDA regs with­out fre­quent re­views

HHS on Thursday finalized its decision to withdraw a rule, proposed just before former President Donald Trump left office, that would’ve caused thousands of HHS and FDA regulations to automatically expire if they weren’t reviewed within two years, and every 10 years thereafter.

The decision follows the filing of a lawsuit last March, in which several nonprofits alleged that the outgoing administration planted “a ticking timebomb” for HHS, essentially forcing it to devote an enormous amount of resources to the unprecedented and infeasible task of reviewing thousands of regulations regularly.

Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

Vi­iV Health­care looks to make long-act­ing HIV pre­ven­tion shot ac­ces­si­ble in low- and mid­dle-in­come coun­tries

The Joint United Nations Programme on HIV and AIDS set a lofty goal back in 2019 to end the HIV epidemic by 2030. But according to the World Health Organization, infection rates are not falling rapidly enough to meet that target.

GSK’s ViiV Healthcare thinks it can help change that.

On Friday, ViiV announced that it’s in talks with the UN-backed Medicines Patent Pool (MPP) for patent rights to its cabotegravir long-acting HIV injectable for pre-exposure prophylaxis (PrEP) in low- and middle-income countries.